Clinical Characteristics and Treatment Outcome of the Desmoplastic Small Round Cell Tumor / 임상소아혈액종양

ABSTRACT

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is an aggressive malignancy with a poor prognosis. DSRCT is a rare disease, and therefore a standard treatment regimen has not been established. In this study, we reviewed the clinical characteristics and treatment outcomes of pediatric DSRCT patients.METHODS: We retrospectively reviewed the medical records of 5 DSRCT patients (2 boys, 3 girls) that were diagnosed and treated with DSRCT at Seoul National University Children's Hospital from January 1999 to January 2015.RESULTS: The median age at diagnosis was 11 years 5months (range 4 years 10 months-17 years 2 months). The most frequent symptoms were abdominal pain (60%). The primary sites were gastrointestinal tract, bladder, and omentum, and the involved sites were the liver, gastrointestinal tract, bladder and bone. Three patients had multiple metastases at diagnosis. Two patients underwent upfront surgical excision of primary tumor, and the remaining 3 patients received neo-adjuvant chemotherapy after the diagnosis was confirmed by using needle biopsy. Combination chemotherapy was administered to all patients in addition to radiotherapy (median dose 45 Gy, range 17.5-54 Gy). Four patients showed disease progression or relapse, resulting in a 20% overall survival rate. At the time of analysis, one patient is alive. She had localized disease at the time of diagnosis and were treated with upfront surgery, chemotherapy, and high-dose chemotherapy with autologous stem cell transplantation and radiotherapy.CONCLUSION: Patients with DSRCT have a poor prognosis, even after multimodal treatment. Further studies are needed to determine the prognostic factors of DSRCT.